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Meta-Analysis
. 2009 Dec;147(5):752-9.
doi: 10.1111/j.1365-2141.2009.07908.x. Epub 2009 Sep 18.

Efficacy and safety of deferasirox doses of >30 mg/kg per d in patients with transfusion-dependent anaemia and iron overload

Ali Taher  1 , Maria D Cappellini, Elliott Vichinsky, Renzo Galanello, Antonio Piga, Tomasz Lawniczek, Joan Clark, Dany Habr, John B Porter
Affiliations
Free PMC article
Meta-Analysis

Efficacy and safety of deferasirox doses of >30 mg/kg per d in patients with transfusion-dependent anaemia and iron overload

Ali Taher et al. Br J Haematol. 2009 Dec.
Free PMC article

Abstract

The highest approved dose of deferasirox is currently 30 mg/kg per d in many countries; however, some patients require escalation above 30 mg/kg per d to achieve their therapeutic goals. This retrospective analysis investigated the efficacy (based on change in serum ferritin levels) and safety of deferasirox >30 mg/kg per d in adult and paediatric patients with transfusion-dependent anaemias, including beta-thalassaemia, sickle cell disease and the myelodysplastic syndromes. In total, 264 patients pooled from four clinical trials received doses of >30 mg/kg per d; median exposure to deferasirox >30 mg/kg per d was 36 weeks. In the overall population there was a statistically significant median decrease in serum ferritin of 440 microg/l (P < 0.0001) from pre-dose-escalation to the time-of-analysis; significant decreases were also observed in adult and paediatric patients, as well as beta-thalassaemia patients. The adverse event profile in patients who received deferasirox doses of >30 mg/kg per d was consistent with previously published data. There was no worsening of renal or liver function following dose escalation. Deferasirox >30 mg/kg per d effectively reduced iron burden to levels lower than those achieved prior to dose escalation in patients with transfusion-dependent anaemias. This has important implications for patients who are heavily transfused and may require higher doses to reduce body iron burden.

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Figures

Fig 1
Fig 1
Relative change (%) in serum ferritin levels from pre-dose escalation (efficacy population). Note: The boxes represent the 25th and 75th percentiles, while the whiskers correspond to the 10th and 90th percentiles. The medians are connected.
Fig 2
Fig 2
Serum creatinine levels before and after dose escalation to doses >30 mg/kg per d (safety population). Note: The boxes represent the 25th and 75th percentiles, while the whiskers correspond to the 10th and 90th percentiles. The medians are connected.
See this image and copyright information in PMC

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